Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Pharmacogenetics of VOD in Children With HSCT (MVO)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03664427
Recruitment Status : Recruiting
First Posted : September 10, 2018
Last Update Posted : February 5, 2019
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:

This project aims to identify common pharmacogenetic biomarkers predisposing children with cancer to develop hepatic VOD during their cancer treatment including HSCT. The impact of VOD occurrence and significant biomarkers will also be evaluated on outcome at day 100 and one year after HSCT. It should help to highlight factors that can contribute to the initiation of hepatic VOD.

Understanding mechanisms of this toxicity and to know individual parameters of disease susceptibility becomes an important issue in the care of these children. The ultimate goal of research in this area would be to develop a personalized predictive medicine and, hopefully, prevent the occurrence of VOD from a therapeutic adaptation to each patient according to his pharmacogenetic profile (adapted prophylaxis, dose adjustment, drug combinations ...). A prospective identification of patients at risk of hepatic VOD will increase the safe use of anticancer.


Condition or disease
Cancer

Detailed Description:
Hematopoietic stem cells transplantation (HSCT) in children with cancer is source of veno-occlusive disease (VOD). This complication is unpredictable and serious by involving the vital prognosis of the child. In addition, this complication may affect the patient's quality of life and have serious long-term sequelae. The incidence varies from 15 to 60% and the mortality is greater than 60% after severe VOD. The risk factors of occurrence of these complications are, to date, unknown except for a susceptibility to some therapeutic (busulfan, radiotherapy ...). Pharmacogenetic aspects of hepatic VOD susceptibility are supposed and targeted screening supported this hypothesis. But pharmacogenetic predisposition to VOD was never explored with as many polymorphisms and considering the whole exome.

Layout table for study information
Study Type : Observational
Estimated Enrollment : 436 participants
Observational Model: Case-Control
Time Perspective: Retrospective
Official Title: Pharmacogenetics of Veno-Occlusive Disease (VOD) in Children With Haematological Stem Cell Transplantation (HSCT)
Actual Study Start Date : January 15, 2019
Estimated Primary Completion Date : January 15, 2020
Estimated Study Completion Date : January 15, 2021

Group/Cohort
Group 1: Patients with VOD
paediatric patients with Veno-Occlusive Disease (VOD) complicating haematological stem cell transplantation
Group 2: matched controls
Paediatric patients defined as matched controls without veno-occlusive disease complicating haematological stem cell transplantation



Primary Outcome Measures :
  1. Pharmacogenetic biomarkers [ Time Frame: 12 months ]
    The pharmacogenetic analysis will be conducted by a whole exome genotyping approach with Microarrays Illumina "Human Omni2.5-8 v1.3" (exploring more than 2,600,000 genetic variants covering the entire genome with more than 300,000 genetic biomarkers within exons).


Secondary Outcome Measures :
  1. Survival status at 100 days post HSCT [ Time Frame: 100 days ]
    Survival at 100 days post-HSCT will be evaluated according to the occurrence or not of VOD

  2. Survival status at 1 year post HSCT [ Time Frame: 12 months ]
    Survival at 1 year post-HSCT will be evaluated according to the occurrence or not of VOD



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   6 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
This study concerns children with cancer aged less than 18 years treated for their first HSCT between 2000 and 2011 in France.
Criteria

Inclusion Criteria:

  • Children with cancer aged less than 18 years old treated for their first HSCT between 2000 and 2011 in France.
  • Patients are selected from the database ProMise regarding pediatric patients treated in any center of the French Society of Stem Cell transplantation (SFGM).
  • Clinical data (age, sex, initial pathology, conditioning treatment, type of graft cells, VOD occurence or not, survival status at 100 days and 1 year after transplantation) were extracted from this database.

Exclusion Criteria:

-


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03664427


Contacts
Layout table for location contacts
Contact: Evelyne Jacqz-Aigrain, MD, PhD +33(0)140032150 evelyne.jacqz-aigrain@aphp.fr
Contact: Jean-Hugues Dalle, MD, PhD +33 (0) 40 03 12 41 jean-hugues.dalle@aphp.fr

Locations
Layout table for location information
France
Robert Debre Hospital Recruiting
Paris, France, 75019
Contact: Tiphaine Debeaumais, PharmD, PhD    +33(0)140032150    tiphaine.debeaumais@gmail.com   
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris

Layout table for additonal information
Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03664427     History of Changes
Other Study ID Numbers: NI16025J
First Posted: September 10, 2018    Key Record Dates
Last Update Posted: February 5, 2019
Last Verified: July 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No